{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T07:01:27Z","timestamp":1790406087976,"version":"4.1.0"},"reference-count":92,"publisher":"Oxford University Press (OUP)","license":[{"start":{"date-parts":[[2020,6,10]],"date-time":"2020-06-10T00:00:00Z","timestamp":1591747200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000897","name":"Cystic Fibrosis Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000897","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, encoding an anion channel that conducts chloride and bicarbonate across epithelial membranes. Mutations that disrupt pre-mRNA splicing occur in &amp;gt;15% of CF cases. One common CFTR splicing mutation is CFTR c.3718-2477C&amp;gt;T (3849+10\u00a0kb C&amp;gt;T), which creates a new 5\u2032 splice site, resulting in splicing to a cryptic exon with a premature termination codon. Splice-switching antisense oligonucleotides (ASOs) have emerged as an effective therapeutic strategy to block aberrant splicing. We test an ASO targeting the CFTR c.3718-2477C&amp;gt;T mutation and show that it effectively blocks aberrant splicing in primary bronchial epithelial (hBE) cells from CF patients with the mutation. ASO treatment results in long-term improvement in CFTR activity in hBE cells, as demonstrated by a recovery of chloride secretion and apical membrane conductance. We also show that the ASO is more effective at recovering chloride secretion in our assay than ivacaftor, the potentiator treatment currently available to these patients. Our findings demonstrate the utility of ASOs in correcting CFTR expression and channel activity in a manner expected to be therapeutic in patients.<\/jats:p>","DOI":"10.1093\/nar\/gkaa490","type":"journal-article","created":{"date-parts":[[2020,6,5]],"date-time":"2020-06-05T15:26:03Z","timestamp":1591370763000},"source":"Crossref","is-referenced-by-count":14,"title":["Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells"],"prefix":"10.1093","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1928-7956","authenticated-orcid":false,"given":"Wren E","family":"Michaels","sequence":"first","affiliation":[{"name":"Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL\u00a060064, USA"},{"name":"School of Graduate and Postdoctoral Studies, Rosalind Franklin University of Medicine and Science, North Chicago, IL\u00a060064, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert J","family":"Bridges","sequence":"first","affiliation":[{"name":"Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL\u00a060064, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michelle L","family":"Hastings","sequence":"first","affiliation":[{"name":"Center for Genetic Diseases, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL\u00a060064, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2020,6,10]]},"reference":[{"key":"2020061011343450000_B1","doi-asserted-by":"crossref","first-page":"1066","DOI":"10.1126\/science.2475911","article-title":"Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA","volume":"245","author":"Riordan","year":"1989","journal-title":"Science"},{"key":"2020061011343450000_B2","doi-asserted-by":"crossref","first-page":"93","DOI":"10.1007\/s00018-016-2391-y","article-title":"Role of CFTR in epithelial physiology","volume":"74","author":"Saint-Criq","year":"2017","journal-title":"Cell. 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